MICA: Childhood Arthritis Response to Treatment consortium - partnership to define stratified medicine tools for childhood inflammatory arthritis

关节炎(关节肿胀、疼痛和炎症)影响每1000名儿童和年轻人中的1人。儿童和青少年的关节炎被称为幼年特发性关节炎(JIA)。如果JIA没有得到充分的治疗，它会导致剧烈疼痛、关节受限肿胀和僵硬、疲劳、生长不良、骨骼变薄、缺课、社交和体育活动减少以及失去家庭生活机会。贾樟柯可能会导致严重的残疾，这种残疾会持续到成年。有许多现代的、有效的药物可以用来比以前更好地治疗JIA。然而，我们还没有可靠的、对儿童友好的测试来预测哪种药物对每个儿童最有效。这些药物中的每一种都可能有严重的副作用。目前，儿童和年轻人(及其家人)在开始服用一种新药后，必须等待，看看它是否有效，以及是否会出现任何重要的副作用。如果它不起作用，或者出现副作用，只有到那时，他们才被允许尝试另一种方法。这种“让我们试试看”的方法需要时间，并使儿童接触到可能不起作用的药物，但往往会产生重要的短期和长期副作用。因此，迫切需要开发一种方法来帮助预测单个儿童对每种药物的反应，从而使患有JIA的儿童能够得到最合适的药物治疗。这种方法现在被称为“精确”或“分层”医学。早期控制关节炎可以让孩子们恢复他们的教育，以及充分的体育和家庭生活活动，而继续控制关节炎可以预防残疾，从长远来看避免失去机会。目前，英国四项主要研究正在收集全国范围内患有JIA的儿童和年轻人对治疗反应的有价值的数据。在他们之间，这些研究有大量的数据，包括临床和实验室数据，可以满足这一需求。在这一伙伴关系中，我们建议将这些研究结合在一起，形成一个独特的联盟，称为儿童关节炎治疗反应联盟(图表)。查特将与所有主要的国内和国际利益攸关方合作，包括临床和研究合作者，以及生活受到JIA影响的家庭和患者。CHART的总体目标是直接改善关节炎儿童的临床护理。CHART联盟计划的工作的关键方面是：1.准确地找出英国对JIA2的四项主要研究中已经有哪些数据和样本。设计并商定一种共享所有数据的方法，并将数据集中在一起。3.同意收集数据和样本，以了解未来以同样的方式治疗的反应，以加快所有患有JIA4的儿童的进展。让更多的儿童和家庭有机会通过包括新的中心和合作者来为这些研究做出贡献如果成立，CHART联盟将成为世界上最大的此类联盟之一。图表研究人员将有一个共同的目标，即使JIA的治疗更加个性化和精确，帮助每个关节炎儿童尽快恢复完全正常的生活。

Arthritis (swelling, pain and inflammation of the joints) affects 1 in 1000 children and young people. Arthritis of children and young people is called juvenile idiopathic arthritis (JIA). If JIA is not fully treated it causes severe pain, joint restriction swelling and stiffness, fatigue, poor growth, thinning of bones, time off school, reduced social and sporting activities and lost family life opportunities. JIA can lead to major disability that lasts well into adult life. There are many modern, powerful medicines that can be used to treat JIA better than before. However we do not yet have reliable and child-friendly tests that can predict which drug will work best for each child. Each of these drugs may also have serious side effects. At the moment, children and young people (and their families) have to wait after starting a new drug to see if it works, and whether any important side effects happen. If it does not work, or side effects crop up, only then are they allowed to try another. This "lets try it and see" approach takes time and exposes children to drugs which might not work well, yet often have important short and long term side effects. Therefore there is an urgent need to develop ways that can help to predict how an individual child will respond to each drug, and so allow children with JIA to be treated with the most appropriate medicine. This approach is now known as 'precision' or 'stratified' medicine. Early control of the arthritis allows children to return to their education, as well as full sporting and family life activities, whilst continued control of arthritis prevents disability and avoids lost opportunities in the long-term.At the moment, four major UK studies are collecting valuable data about response to treatment from children and young people with JIA across the country. Between them, these studies have a lot of data, both clinical as well as laboratory data, that can address this need. In this partnership we propose to bring these studies together to form a unique Consortium, called the Childhood Arthritis Response to Treatment Consortium (CHART). CHART will work with all the main national and international stakeholders including clinical and research collaborators, as well as families and patients who lives are affected by JIA. The overall aim of CHART is to directly improve clinical care for children with arthritis. The key aspects of the work planned in the CHART Consortium are to : 1. Find out exactly what data and samples are already available in the four major UK studies of JIA 2. Design and agree a way to share all of the data, and bring the data together 3. Agree to collect data and samples for understanding response to treatment in the same way in the future, in order to speed up progress for all children with JIA4. Give many more children and families the opportunity to contribute to these studies by including new centres and collaboratorsThe CHART Consortium, if established, will be one of the largest of its kind in the world. The CHART researchers will have the common goal of making treatment of JIA more individualised and precise, to help every child with arthritis return to a full normal life as quickly as possible.